Use of JNK Signaling and JNK Inhibitors for the Treatment of Spinal Muscular Atrophy

Case ID:

2009-036

Web Published:

7/26/2011

Description:

Current
State of the Art:

SMA
is a devastating neurodegenerative disorder with progressive muscle weakness and
loss of motor control due to the loss of neurons in the spinal cord. SMA affects
approximately 4 out of every 100,000 people. Children born with SMA Type I, die
within 2 years of birth. Those diagnosed with Type II and III can live on into
adulthood with strictly managed care.

Drugs
currently being used off-label to treat SMA include Albuterol, Hyrdoxyurea,
Riluzole, Somatotropin, Sodium Phenylbutyrate and Valproic
Acid.

Disadvantages
with the Current Art:

Therapeutics
are not readily available for SMA that have been shown to treat the debilitating
and sometimes lethal disease. Patients are offered off-label use of FDA approved
drugs while other targeted therapies await FDA approval.

Advantages
of Invention:

The
inventor has identified a promising therapeutic regimen that would prevent loss
of motor neurons in the spinal cord of an animal model system for neural
degeneration: